Posts by: Joana Carvalho

Joana Carvalho

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

February 26, 2021February 26, 2021

FDA Partially Rescinds Indications for BeneFIX and Ixinity

News

Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially rescinded its decision to approve BeneFIX and Ixinity for additional indications both therapies received last year ... Read more

February 19, 2021February 19, 2021

Efanesoctocog Alfa on Fast Track for Hemophilia A

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s ... Read more

February 15, 2021February 15, 2021

Preventive Advate Found Better Than On-demand Treatment in Hemophilia A

News

When given as a prophylactic, or preventive therapy, Advate worked better than on-demand treatment at lowering the number of spontaneous bleeds in people with moderate or severe hemophilia A, according ... Read more

February 8, 2021February 11, 2021

Gene Therapy SPK-8016 Shows Early Promise for Severe Hemophilia A

News

A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia ... Read more

January 8, 2021January 8, 2021

Hemlibra Lowered Number of Hemophilia A Bleeds Over Nearly 6 Years

News

When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data ... Read more

January 4, 2021January 4, 2021

FLT180a Gene Therapy Can Prevent Bleeds in Hemophilia B Patients for Up to 3 Years, Data Show

News

A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing bleeds ... Read more

October 14, 2020October 14, 2020

Interviews Reveal Women With Genetic Blood Disorders Share Uncertainties Regarding Diagnosis

News

Women with genetic blood disorders, including those who are carriers of hemophilia A and B, face multiple uncertainties surrounding their diagnosis, according to a recent interview-based study. Many women claimed ... Read more

September 30, 2020November 6, 2020

CRISPR/Cas9 May Be Better Than Gene Therapy at Restoring FIX Activity

News

Note: This story was updated Nov. 6, 2020, to clarify key scientific concepts. The gene-editing tool CRISPR/Cas9 enables a targeted gene insertion approach that may be superior to conventional gene ... Read more

September 18, 2020September 18, 2020

Roche Proposes Transfer Needle With Filter When Using Hemlibra

News

Reports of translucent particles of being detected in Hemlibra (emicizumab) in 2019 do not pose a safety risk or affect the therapy’s effectiveness and, according to Roche, health authorities have ... Read more